EP4263574A4 - Aav-kapside und vektoren - Google Patents

Aav-kapside und vektoren

Info

Publication number
EP4263574A4
EP4263574A4 EP21904657.0A EP21904657A EP4263574A4 EP 4263574 A4 EP4263574 A4 EP 4263574A4 EP 21904657 A EP21904657 A EP 21904657A EP 4263574 A4 EP4263574 A4 EP 4263574A4
Authority
EP
European Patent Office
Prior art keywords
vectors
aav capsids
capsids
aav
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP21904657.0A
Other languages
English (en)
French (fr)
Other versions
EP4263574A1 (de
Inventor
Adrian Westhaus
Leszek Lisowski
Robyn Jamieson
Steven Eamegdool
Anai Gonzalez Cordero
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Childrens Medical Research Institute
Sydney Childrens Hospitals Network Randwick and Westmead
Original Assignee
Childrens Medical Research Institute
Sydney Childrens Hospitals Network Randwick and Westmead
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority claimed from AU2020904689A external-priority patent/AU2020904689A0/en
Application filed by Childrens Medical Research Institute, Sydney Childrens Hospitals Network Randwick and Westmead filed Critical Childrens Medical Research Institute
Publication of EP4263574A1 publication Critical patent/EP4263574A1/de
Publication of EP4263574A4 publication Critical patent/EP4263574A4/de
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/075—Adenoviridae
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Immunology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Measurement And Recording Of Electrical Phenomena And Electrical Characteristics Of The Living Body (AREA)
  • Prostheses (AREA)
  • Magnetic Resonance Imaging Apparatus (AREA)
EP21904657.0A 2020-12-16 2021-12-16 Aav-kapside und vektoren Pending EP4263574A4 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
AU2020904689A AU2020904689A0 (en) 2020-12-16 AAV capsids and vectors
PCT/AU2021/051496 WO2022126188A1 (en) 2020-12-16 2021-12-16 Aav capsids and vectors

Publications (2)

Publication Number Publication Date
EP4263574A1 EP4263574A1 (de) 2023-10-25
EP4263574A4 true EP4263574A4 (de) 2025-10-22

Family

ID=82059565

Family Applications (1)

Application Number Title Priority Date Filing Date
EP21904657.0A Pending EP4263574A4 (de) 2020-12-16 2021-12-16 Aav-kapside und vektoren

Country Status (4)

Country Link
US (1) US20240059743A1 (de)
EP (1) EP4263574A4 (de)
AU (1) AU2021399882A1 (de)
WO (1) WO2022126188A1 (de)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4413147A4 (de) 2021-10-08 2025-07-30 Dyno Therapeutics Inc Kapsidvarianten und verfahren zur verwendung davon
WO2024182558A1 (en) * 2023-02-28 2024-09-06 The Saban Research Institute; Children's Hospital Los Angeles Fluorescent cone reporter ips cells, retinal organoids and uses thereof
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
EP4688808A1 (de) * 2023-04-04 2026-02-11 Children's Medical Research Institute Aav-kapside und vektoren zur transduktion von zellen des peripheren nervensystems
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2025214359A1 (en) * 2024-04-09 2025-10-16 Hangzhou Jiayin Biotech Ltd. A novel aav variant
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019076856A1 (en) * 2017-10-16 2019-04-25 Vigeneron Gmbh AAV VECTORS

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA44546B1 (fr) * 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
ES2946747T3 (es) * 2017-11-27 2023-07-25 4D Molecular Therapeutics Inc Variantes de cápsides de virus adenoasociado y su utilización para inhibir la angiogénesis
TW202102526A (zh) * 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019076856A1 (en) * 2017-10-16 2019-04-25 Vigeneron Gmbh AAV VECTORS

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
DENIZ DALKARA ET AL: "In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous", SCIENCE TRANSLATIONAL MEDICINE, vol. 5, no. 189, 12 June 2013 (2013-06-12), United States, pages 1 - 11, XP055533378, Retrieved from the Internet <URL:http://stm.sciencemag.org/content/scitransmed/5/189/189ra76.full.pdf> DOI: 10.1126/scitranslmed.3005708 *
GARITA-HERNANDEZ MARCELA ET AL: "AAV-Mediated Gene Delivery to 3D Retinal Organoids Derived from Human Induced Pluripotent Stem Cells", INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, vol. 21, no. 3, 3 February 2020 (2020-02-03), pages 994, XP093030213, DOI: 10.3390/ijms21030994 *
KEN Y CHAN ET AL: "Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems", NATURE NEUROSCIENCE, vol. 20, no. 8, 26 June 2017 (2017-06-26), New York, pages 1172 - 1179, XP055527909, ISSN: 1097-6256, DOI: 10.1038/nn.4593 *
See also references of WO2022126188A1 *
WESTHAUS ADRIAN ET AL: "AAV capsid bioengineering in primary human retina models", SCIENTIFIC REPORTS, vol. 13, no. 1, 11 December 2023 (2023-12-11), US, XP093266292, ISSN: 2045-2322, Retrieved from the Internet <URL:https://www.nature.com/articles/s41598-023-49112-2> DOI: 10.1038/s41598-023-49112-2 *
WESTHAUS ADRIAN ET AL: "High-Throughput In Vitro , Ex Vivo, and In Vivo Screen of Adeno-Associated Virus Vectors Based on Physical and Functional Transduction", HUMAN GENE THERAPY, vol. 31, no. 9-10, 1 May 2020 (2020-05-01), GB, pages 575 - 589, XP055885822, ISSN: 1043-0342, Retrieved from the Internet <URL:https://www.liebertpub.com/doi/pdfplus/10.1089/hum.2019.264> DOI: 10.1089/hum.2019.264 *
WESTHAUS ADRIAN: "Supplemental Information: AAV capsid bioengineering in primary human retina models", 11 December 2023 (2023-12-11), XP093266303, Retrieved from the Internet <URL:https://www.nature.com/articles/s41598-023-49112-2#Sec30> *

Also Published As

Publication number Publication date
US20240059743A1 (en) 2024-02-22
WO2022126188A1 (en) 2022-06-23
EP4263574A1 (de) 2023-10-25
AU2021399882A1 (en) 2023-07-06

Similar Documents

Publication Publication Date Title
EP4041755A4 (de) Modifizierte aav-kapside und verwendungen davon
EP3589277A4 (de) Modifizierte aav-kapside und verwendungen davon
EP4122946A4 (de) Adeno-assoziierter virusvektor und verwendung davon
IL307957A (en) AAV capsids and uses thereof
IL282885A (en) Aav viral vectors and uses thereof
IL311861A (en) Aav capsid variants and uses thereof
EP4263575A4 (de) Kapside und vektoren von adeno-assoziiertem virus
EP3726122A4 (de) Stabilisatorfaltanordnung und handstabilisator
PL3740222T3 (pl) Zmodyfikowane białko kapsydu raav do terapii genowej
EP3528785A4 (de) Modifizierte aav-kaspide und verwendungen davon
DK3684423T5 (da) Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf
EP3955891C0 (de) Adeno-assoziierte virusvektorformulierungen und verfahren
EP3689771C0 (de) Tülle für eine verpackung und verpackung-tülle-anordnung
EP4442698A4 (de) Modifiziertes aav-kapsidprotein und verwendung davon
EP4392436A4 (de) Modifizierte aav-kapside und vektoren
EP4288112A4 (de) Rna-adeno-assoziierter virus (raav)-vektor und verwendungen davon
EP4019642A4 (de) Isoliertes modifiziertes vp1-kapsidprotein von aav5
PL3999119T3 (pl) Modyfikowane białka kapsydu aav do leczenia choroby zapalnej stawów
EP3964531A4 (de) Protein-molekül und verwendung davon
EP4272751C0 (de) Compstatin-analoga und deren medizinische verwendungen
EP4395723A4 (de) Temperaturgesteuerte und vibrierende therapeutische bandanordnung
EP4387641A4 (de) Rekombinante aav-formulierungen
EP3723771A4 (de) Rekombinante adenoviren und verwendungen davon
EP3858866A4 (de) Glp1-fc-fusionsprotein und konjugat davon
EP4452934A4 (de) Ionisierbare disulfidlipide und daraus gewonnene lipidnanopartikel

Legal Events

Date Code Title Description
STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: THE INTERNATIONAL PUBLICATION HAS BEEN MADE

PUAI Public reference made under article 153(3) epc to a published international application that has entered the european phase

Free format text: ORIGINAL CODE: 0009012

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: REQUEST FOR EXAMINATION WAS MADE

17P Request for examination filed

Effective date: 20230717

AK Designated contracting states

Kind code of ref document: A1

Designated state(s): AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR

DAV Request for validation of the european patent (deleted)
DAX Request for extension of the european patent (deleted)
RIC1 Information provided on ipc code assigned before grant

Ipc: C12N 7/00 20060101ALI20250506BHEP

Ipc: C12N 15/86 20060101ALI20250506BHEP

Ipc: C07K 14/005 20060101AFI20250506BHEP

A4 Supplementary search report drawn up and despatched

Effective date: 20250917

RIC1 Information provided on ipc code assigned before grant

Ipc: C07K 14/005 20060101AFI20250912BHEP

Ipc: C12N 15/86 20060101ALI20250912BHEP

Ipc: C12N 7/00 20060101ALI20250912BHEP